ELK GROVE VILLAGE, Ill. and SOUTH SAN FRANCISCO, Calif., May 17, 2018 (GLOBE NEWSWIRE) — Cure SMA and Cytokineticstoday announced an expanded partnership to increase education, awareness and fundraising for spinal muscular atrophy (SMA). As a National Platinum Partner for 2018, Cytokineticswill lend support to several of Cure SMA’s initiatives at both the local and national level to advance understanding of, and research toward potential treatments for SMA, a genetic disease that robs people of physical strength and takes away their ability to walk, eat or breathe.
“We have enjoyed a longstanding relationship with Cure SMA and are pleased to expand our involvements, particularly at a time when more attention and research are focused on the SMA community,” said Robert I. Blum, President and Chief Executive Officer of Cytokinetics. “We remain committed to advancing therapies for adolescents and adults with types II, III and IV SMA, a population in need of new therapies to address impaired muscle function and weakness despite the promise of gene-directed therapies.”
On Thursday, April 26, Cure SMA traveled with more than 70 spinal muscular atrophy advocates to Washington D.C. to meet with over 60 congressional offices. The advocates represented 24 states and D.C. The purpose of the advocacy day was to advance life-changing opportunities for our community through SMA research and newborn screening.
and this is a awesome news for more than 300 families living with SMA in Australia.
Health Minister Greg Hunt told the ABC from June 2018, Spinraza will be available on the Pharmaceutical Benefits Scheme (PBS) for the treatment of Type 1, Type 2 and Type 3a SMA for all patients under the age of 18.
”This will be both life-saving and life-changing for hundreds of young patients and their families,” he said.
Researchers at The Ottawa Hospital and the University of Ottawa have discovered a completely new function for the protein complex containing the dystrophin protein, which is missing in people with Duchenne muscular dystrophy